Adapting to Adaptive Licensing

Lynn Baird referred to adaptive licensing as the “future of licensing” in the November issue of The Medicine Maker (“Adapting to the Future of Licensing” [tmm.txp.to/0214/adapting]) and it seems that many companies feel the same way. A recent progress report released by the European Medicines Agency (EMA) shows that 34 submissions had been received as of December 2014 for its adaptive licensing pilot project – Adaptive Pathways.

Adaptive licensing has a more staggered approval process, with the medicine first authorized in a restricted patient population under close monitoring. If all goes well, the marketing authorization can gradually be expanded to new patient populations, with data being accumulated along the way.

The majority of the requests received by the EMA related to anticancer medicines and orphan medicines, and six submissions have now been selected for what the EMA refers to as a ‘Stage II meeting’, which involves an in-depth discussion with all stakeholders. One such meeting has already taken place and the others are planned for 2015.

Lynn Baird referred to adaptive licensing as the “future of licensing” in the November issue of The Medicine Maker (“Adapting to the Future of Licensing” [tmm.txp.to/0214/adapting]) and it seems that many companies feel the same way. A recent progress report released by the European Medicines Agency (EMA) shows that 34 submissions had been received as of December 2014 for its adaptive licensing pilot project – Adaptive Pathways.

Adaptive licensing has a more staggered approval process, with the medicine first authorized in a restricted patient population under close monitoring. If all goes well, the marketing authorization can gradually be expanded to new patient populations, with data being accumulated along the way.

The majority of the requests received by the EMA related to anticancer medicines and orphan medicines, and six submissions have now been selected for what the EMA refers to as a ‘Stage II meeting’, which involves an in-depth discussion with all stakeholders. One such meeting has already taken place and the others are planned for 2015.

After February 28, the EMA will only accept new submissions that are “well developed” and hold potential for “meaningful” Stage II meetings. Specifically, the agency is looking for submissions that are amenable to health technology assessment (HTA) and a real-world data approach, and that carefully consider the indications and populations that would be suitable for initial use of the product. The EMA also handed out additional advice for applicants: don’t be vague.

“Applicants are reminded that general statements (e.g., ‘a registry will be set up to collect post authorisation data’ or ‘we anticipate a high level of interest from HTAs’) do not provide sufficient elements to evaluate the suitability of an approach for the Adaptive Pathways, and should be avoided when filling the application form,” says the EMA report.  “Scenario-planning, based on ‘what-if’ scenarios, is welcome so that multiple pathways can be discussed,” adds the EMA.

The report summarizes the EMA’s initial experience with the pilot, but a full review of the outcome and impact will be made when at least six products have completed the procedure of parallel scientific advice with HTA bodies.